New gene delivery vehicle shows promise for human brain gene therapy
a day ago
- #Gene Therapy
- #AAV Vector
- #Blood-Brain Barrier
- Engineered adeno-associated virus (AAV) crosses human blood-brain barrier efficiently in cell models.
- Delivers genes throughout brain in humanized mice, showing promise for brain gene therapy.
- Funding from multiple organizations including Apertura Gene Therapy and NIH.
- Research published in Science on May 16, 2024.